Prospective Multisite Study of Quality of Life in Pediatric Intestinal Failure

Pediatric Intestinal Failure Clinical Trial

Official title:

Prospective Multisite Study of Quality of Life in Pediatric Intestinal Failure

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04629014
• Other study ID #: P00032389
• Status: Recruiting
• Start date: February 27, 2020
• Est. completion date: December 2025

Study information

• Verified date: January 2024
• Source: Boston Children's Hospital
• Contact: Biren P Modi, MD MPH
• Phone: 857-218-4651
• Email: biren.modi[@]childrens.harvard.edu
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This study proposes to quantify and describe the quality of life of children with intestinal failure, and to identify the medical and socio-economic factors that impact this quality of life, using data from multiple multidisciplinary intestinal failure centers across the United States and Canada specializing in the care of these participants.

Description:

Health related quality of life is becoming an increasingly important topic as medical advancements continue to increase life expectancies in various illnesses. Quality of life (QoL) encompasses the physical, psychological, and social well-being of a person. Studies focusing on health related QoL show that providers have a limited understanding of the QoL of their patients, demonstrating a need for further research. Mortality rates in pediatric intestinal failure (PIF) have significantly dropped over the past 3-4 decades and long-term morbidity rather than survival has come into focus as the outcome of interest. However, there remains a significant knowledge gap regarding the specific factors that contribute to QoL among PIF patients and their families. Insight into the challenges these patients face can promote enhanced support services and psycho-education for caregivers of these children as well as help to tailor medical and surgical treatment with a focus on improved QoL. Due to the rare nature of PIF, and despite a recent bloom in multidisciplinary PIF centers, experience is limited in individual centers and a multi-site study with a robust and diverse patient population is optimal to study QoL in pediatric intestinal failure.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 400
• Est. completion date: December 2025
• Est. primary completion date: December 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 25 Years

Eligibility

Inclusion Criteria:

• Participants will be followed in a participating institution's intestinal rehabilitation program

• Participants will have a diagnosis of intestinal failure due to functional or structural intestinal dysfunction with current or prior history of specialized nutritional support (parenteral nutrition requirement for 60 out of 74 consecutive days)

• Participants will be age 6 months to 25 years old.

• Parents/caregivers must be able to complete questionnaire without assistance.

• English or Spanish speaking

Exclusion Criteria:

• Participants aged less than 6 months or greater than 25 years will not be included in this study

• Participants will not be enrolled less than 3 months from index admission or initial outpatient evaluation

• Participants will not be enrolled less than 1 month from inpatient admission (any admission greater than 24 hours in duration)

• Participants will not be enrolled less than 1 month from any operative intervention requiring general anesthesia.

• Primary language other than English or Spanish.

• Participants who have a currently functional small bowel, liver/small bowel or multivisceral transplant

• While other major co-morbidities may be excluded at a later time during data analysis, or may be analyzed as a specific sub-group, they will not be excluded outright. For example, participants with global developmental delay may have parent surveys only, and therefore would need to be excluded from paired analysis of proxy vs. participant perception of HRQOL

Related Conditions & MeSH terms

• Intestinal Failure
• Pediatric Intestinal Failure

Locations

Country	Name	City	State
Canada	Alberta Children's Hospital	Calgary	Alberta
Canada	Hospital for Sick Children	Toronto	Ontario
Canada	BC Children's Hospital	Vancouver	British Columbia
United States	C.S. Mott Children's Hospital	Ann Arbor	Michigan
United States	Children's of Alabama	Birmingham	Alabama
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Lurie Children's Hospital	Chicago	Illinois
United States	Dallas Children's Hospital	Dallas	Texas
United States	Duke Children's Hospital	Durham	North Carolina
United States	St. Louis Children's Hospital	Saint Louis	Missouri
United States	Seattle Children's Hospital	Seattle	Washington

Sponsors (11)

Lead Sponsor	Collaborator
Boston Children's Hospital	Alberta Children's Hospital, Ann & Robert H Lurie Children's Hospital of Chicago, British Columbia Children's Hospital, Duke Health, Seattle Children's Hospital, The Hospital for Sick Children, University of Alabama at Birmingham, University of Michigan, University of Texas Southwestern Medical Center, Washington University School of Medicine

Countries where clinical trial is conducted

United States,  Canada, 

References & Publications (7)

Diamond IR, de Silva N, Pencharz PB, Kim JH, Wales PW; Group for the Improvement of Intestinal Function and Treatment. Neonatal short bowel syndrome outcomes after the establishment of the first Canadian multidisciplinary intestinal rehabilitation program: preliminary experience. J Pediatr Surg. 2007 May;42(5):806-11. doi: 10.1016/j.jpedsurg.2006.12.033. — View Citation

Javid PJ, Malone FR, Reyes J, Healey PJ, Horslen SP. The experience of a regional pediatric intestinal failure program: Successful outcomes from intestinal rehabilitation. Am J Surg. 2010 May;199(5):676-9. doi: 10.1016/j.amjsurg.2010.01.013. — View Citation

Modi BP, Langer M, Ching YA, Valim C, Waterford SD, Iglesias J, Duro D, Lo C, Jaksic T, Duggan C. Improved survival in a multidisciplinary short bowel syndrome program. J Pediatr Surg. 2008 Jan;43(1):20-4. doi: 10.1016/j.jpedsurg.2007.09.014. — View Citation

Mutanen A, Kosola S, Merras-Salmio L, Kolho KL, Pakarinen MP. Long-term health-related quality of life of patients with pediatric onset intestinal failure. J Pediatr Surg. 2015 Nov;50(11):1854-8. doi: 10.1016/j.jpedsurg.2015.05.012. Epub 2015 Jun 3. — View Citation

Norsa L, Artru S, Lambe C, Talbotec C, Pigneur B, Ruemmele F, Colomb V, Capito C, Chardot C, Lacaille F, Goulet O. Long term outcomes of intestinal rehabilitation in children with neonatal very short bowel syndrome: Parenteral nutrition or intestinal transplantation. Clin Nutr. 2019 Apr;38(2):926-933. doi: 10.1016/j.clnu.2018.02.004. Epub 2018 Feb 15. — View Citation

Sanchez SE, McAteer JP, Goldin AB, Horslen S, Huebner CE, Javid PJ. Health-related quality of life in children with intestinal failure. J Pediatr Gastroenterol Nutr. 2013 Sep;57(3):330-4. doi: 10.1097/MPG.0b013e3182999961. — View Citation

Varni JW, Limbers CA, Burwinkle TM. Impaired health-related quality of life in children and adolescents with chronic conditions: a comparative analysis of 10 disease clusters and 33 disease categories/severities utilizing the PedsQL 4.0 Generic Core Scales. Health Qual Life Outcomes. 2007 Jul 16;5:43. doi: 10.1186/1477-7525-5-43. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Health-related quality of life (hrQOL)	hrQOL as determined by PedsQL Generic Core at time of enrollment and corrected for time from onset of disease	Baseline
Primary	Health-related quality of life (hrQOL) Change over time	hrQOL as determined by PedsQL Generic Core, assessed annually, looking at trend in change over 5 years of assessment and corrected for time from onset of disease	Trend over 5 years
Primary	Disease-specific Health-related quality of life (hrQOL)	Disease-specific hrQOL as determined by PedsQL GI Symptoms Scale at time of enrollment and corrected for time from onset of disease	Baseline
Primary	Disease-specific Health-related quality of life (hrQOL)	Disease-specific hrQOL as determined by PedsQL GI Symptoms Scale, assessed annually, looking at trend in change over 5 years of assessment and corrected for time from onset of disease	Trend over 5 years
Secondary	Family Impact	Family impact as determined by the PedsQL Family Impact Module	Baseline
Secondary	Family Impact	Family impact as assessed by the PedsQL Family Impact Module, assessed annually, looking at trend in change over 5 years of assessment and corrected for time from onset of disease	Trend over 5 years